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The Significance of the MHRA Approval and Upcoming FDA Review of the First Gene Editing Treatment

Worldwide Clinical Trials

Casgevy, the commercial product formerly known as exa-cel, is administered by taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a laboratory, with the modified cells then infused back into the patient after conditioning treatment to prepare the bone marrow.

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Sanofi expands research deal with gene editing startup Scribe

Bio Pharma Dive

Scribe will receive $40 million upfront from Sanofi in a collaboration initially focused on developing an in vivo gene editing treatment for sickle cell disease.

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Gene editing biotech Homology hits reset and seeks a sale

Bio Pharma Dive

The company will lay off 87% of its workforce and halt further research, including for a gene editing therapy to treat the rare disease PKU.

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Pfizer expands into gene editing with Beam research deal

Bio Pharma Dive

Pfizer's work with mRNA vaccines led it explore other applications of the technology, resulting in a multi-year partnership with the high-profile biotech Beam Therapeutics on gene editing treatments for rare diseases.

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FDA clears Intellia to start US tests of ‘in vivo’ gene editing drug

Bio Pharma Dive

The announcement, which follows recent regulatory setbacks for some of Intellia’s peers, is a “big win” for the gene editing field, according to one analyst.

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Vertex aligns with another CRISPR gene editing startup

Bio Pharma Dive

A new alliance with Mammoth Biosciences marks the latest evidence of Vertex's interest in gene editing, which has already been the focus of multiple deals with emerging biotechs.

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FDA sets out advice to developers of gene editing medicines

Bio Pharma Dive

Draft recommendations from the agency were generally straightforward, analysts said, although the document emphasized the potential safety risks of gene editing.